PTC Provides Update on Translarna™ Application for Label Expansion

PTC Therapeutics announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has completed their review of a proposed indication extension of Translarna™ (ataluren) for the treatment of patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) who are non-ambulatory. While the CHMP adopted a negative opinion of the extension, PTC was informed by EMA representatives that the European Public Assessment Report (EPAR) will be updated to clarify that patients who start Translarna while ambulatory are not required to discontinue treatment after loss of ambulation.

Translarna is currently indicated for ambulatory Duchenne patients who are over two years of age; the requested extension would have allowed for the inclusion of non-ambulatory patients in the label. PTC plans to request a re-examination of the procedure within the next two weeks and expects the new examination to last approximately 4 months.

"While we are disappointed with the current outcome of the label expansion procedure and its impact on non-ambulatory patients with nonsense mutation Duchenne Muscular Dystrophy, we are pleased that patients on Translarna can remain on treatment after loss of ambulation," said Marcio Souza, Chief Operating Officer, PTC Therapeutics, Inc. "We remain committed to work with the CHMP to clarify the open questions and are confident we will be able to demonstrate the pulmonary benefit of Translarna in non-ambulatory patients."

The clinical data supporting the extension is based on results supporting the positive impact in the Force Vital Capacity (FVC) parameters for patients treated with Translarna in study 019, a long-term, open-label study, and study 025 (STRIDE Registry) when compared to matched natural history controls. This is in addition to currently approved labeling stating that the pharmacokinetics (PK) and safety profiles are comparable between ambulatory and non-ambulatory nmDMD patients, and that no dose adjustment is necessary when patients become non-ambulatory. The CHMP opined that the comparable PK may not ensure efficacy in non-ambulatory patients since muscle mass is reduced in this patient group. PTC and members of the scientific community expressed disagreement with this understanding during the oral explanation.

Translarna received the annual renewal of its conditional marketing authorization in June 2019 for nonsense mutation Duchenne muscular dystrophy patients who are ambulatory and two years and over. In addition, in connection with the June 2019 renewal, PTC's specific obligation for the submission of the results of Study 041, an ongoing clinical trial of ataluren, has been extended to September 2022.

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